Table 1 High-performance liquid... | American Society of Hematology

Table 1

High-performance liquid chromatography analysis before and after transduction in bulk erythroid culture and in pools of erythroid colonies

Genotype	n	β/α ratio untransduced	β/α ratio transduced	Δ(β/α) ratio	Fold increase	VCN	Δ(β/α)/VCN
Bulk
β⁰/β⁰	11	0.02 ± 0.01	0.44 ± 0.03	0.41 ± 0.03	22	1.88 ± 0.16	0.24 ± 0.02
β⁰/β⁺	6	0.12 ± 0.02	0.47 ± 0.03	0.35 ± 0.03	3.9	1.91 ± 0.31	0.22 ± 0.04
β⁺/β⁺	10	0.32 ± 0.02	0.56 ± 0.03	0.23 ± 0.03	1.75	1.55 ± 0.16	0.17 ± 0.03
Average		0.16 ± 0.03*	0.49 ± 0.02*	0.33 ± 0.02	9.2 ± 6.4	1.74 ± 0.11	0.21 ± 0.02
			Output per copy
Normal	3		0.45 ± 0.02
BFU-E pools
β⁰/β⁰	2	0.01 ± 0.01	0.33 ± 0.2	0.32 ± 0.19	33	0.76 ± 0.35	0.39 ± 0.07
β⁰/β⁺	5	0.14 ± 0.02	0.38 ± 0.03	0.23 ± 0.04	2.7	0.67 ± 0.3	0.58 ± 0.16
β⁺/β⁺	7	0.27 ± 0.02	0.54 ± 0.05	0.28 ± 0.04	2	0.64 ± 0.13	0.53 ± 0.13
Average		0.19 ± 0.03**	0.45 ± 0.04**	0.27 ± 0.03	12.5 ± 10.2	0.67 ± 0.12	0.52 ± 0.09
			Output per copy
Normal	2		0.44 ± 0.08

Genotype	n	β/α ratio untransduced	β/α ratio transduced	Δ(β/α) ratio	Fold increase	VCN	Δ(β/α)/VCN
Bulk
β⁰/β⁰	11	0.02 ± 0.01	0.44 ± 0.03	0.41 ± 0.03	22	1.88 ± 0.16	0.24 ± 0.02
β⁰/β⁺	6	0.12 ± 0.02	0.47 ± 0.03	0.35 ± 0.03	3.9	1.91 ± 0.31	0.22 ± 0.04
β⁺/β⁺	10	0.32 ± 0.02	0.56 ± 0.03	0.23 ± 0.03	1.75	1.55 ± 0.16	0.17 ± 0.03
Average		0.16 ± 0.03*	0.49 ± 0.02*	0.33 ± 0.02	9.2 ± 6.4	1.74 ± 0.11	0.21 ± 0.02
			Output per copy
Normal	3		0.45 ± 0.02
BFU-E pools
β⁰/β⁰	2	0.01 ± 0.01	0.33 ± 0.2	0.32 ± 0.19	33	0.76 ± 0.35	0.39 ± 0.07
β⁰/β⁺	5	0.14 ± 0.02	0.38 ± 0.03	0.23 ± 0.04	2.7	0.67 ± 0.3	0.58 ± 0.16
β⁺/β⁺	7	0.27 ± 0.02	0.54 ± 0.05	0.28 ± 0.04	2	0.64 ± 0.13	0.53 ± 0.13
Average		0.19 ± 0.03**	0.45 ± 0.04**	0.27 ± 0.03	12.5 ± 10.2	0.67 ± 0.12	0.52 ± 0.09
			Output per copy
Normal	2		0.44 ± 0.08

The Δ(β/α) ratio is calculated by subtracting the residual expression measured in untransduced controls from their corresponding gene-corrected samples. The Δ(β/α)/VCN is calculated by subtracting the residual expression measured in untransduced controls from their corresponding gene-corrected samples and normalizing to vector copy number. Output per copy: the β/α ratio per allele of a normal genotype. Data represent individually analyzed samples and are expressed as means ± standard error of the mean. *,**P < .0001 (unpaired Student t test).

UT, untransduced; Td, transduced.

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